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Pediatr Blood Cancer ; 71(4): e30903, 2024 Apr.
Artículo en Inglés | MEDLINE | ID: mdl-38321586

RESUMEN

Opsoclonus-myoclonus-ataxia syndrome (OMAS) is an autoimmune central nervous system disorder, primarily manifesting as a paraneoplastic sequalae to neuroblastoma, and characterized by motor disorders and behavioral disturbances. OMAS is typified by aberrant B-cell and T-cell activation. Current treatment involves immunosuppression using corticosteroids, intravenous immunoglobulin, and rituximab. However, these approaches often lead to treatment-related toxicities and symptomatic recurrences with chronic neurocognitive impairment. We treated three children with refractory neuroblastoma-associated OMAS with tacrolimus, a T-cell-targeting calcineurin inhibitor, effectively controlling symptoms within a month and enabling the discontinuation of immunosuppression with minimal side effects. Tacrolimus shows promise as a therapeutic option for refractory OMAS.


Asunto(s)
Neuroblastoma , Trastornos de la Motilidad Ocular , Síndrome de Opsoclonía-Mioclonía , Niño , Humanos , Tacrolimus/uso terapéutico , Trastornos de la Motilidad Ocular/complicaciones , Síndrome de Opsoclonía-Mioclonía/tratamiento farmacológico , Síndrome de Opsoclonía-Mioclonía/etiología , Síndrome de Opsoclonía-Mioclonía/diagnóstico , Neuroblastoma/complicaciones , Neuroblastoma/tratamiento farmacológico , Neuroblastoma/diagnóstico , Ataxia/complicaciones
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